MHRA guidance on the Innovative Licensing and Access Pathway (ILAP) for medicines

Last updated: 28 February 2022

See updates at the end of this post.

The UK MHRA has published guidance on the the Innovative Licensing and Access Pathway (ILAP) for medicines.

  • It aims to accelerate the time to market, facilitating patient access to medicines.
  • These medicines include new chemical entities, biological medicines, new indications and repurposed medicines.
  • The ILAP awards a new “innovative medicine” designation (Innovation Passport), utilises tools from a toolkit and a “Road Map” (Target Development Profile, TDP).

The pathway brings together innovative approaches to support the safe, timely and efficient development of innovative products

About the pathway

The ILAP is open to both commercial and non-commercial developers of medicines. It is operational from 1 January 2021.

It provides opportunities for enhanced regulatory and other stakeholder input including interactions with NICE, SMC, the NHS and other organisations such as the HRA and the NIHR.

The ILAP enables multiple entry points depending on:

  • the stage of development of the product
  • the data available
  • the ambition of the applicant to engage with UK stakeholders
  • the applicant’s appetite for new innovative ways of working

The pathway will allow entry very early, based on non-clinical data, as well as for products with mid-development ‘global’ dossiers. To maximise the benefits, applicants are encouraged to apply early in the development of their products. Products that are towards the end of their development programme are generally not suitable for the ILAP.

The ILAP will not replace the Early Access to Medicines Scheme (EAMS).

Early pipeline discussions with developers are encouraged, and can be conducted e.g. with the MHRA and partners in the context of scientific advice.

Who are the partners in the ILAP pathway?

The permanent partners are:

  • The MHRA
  • The National Institute for Health and Care Excellence (NICE)
  • The Scottish Medicines Consortium (SMC)
  • The All Wales Therapeutics and Toxicology Centre (AWTTC) -from 4 October 2021. Further information available here.

The supporting partners include:

  • NHS England and NHS Improvement
  • Health Research Authority (HRA)
  • National Institute for Health Research (NIHR)
Which products are in scope of the ILAP?

Medicines in scope of the ILAP include new chemical entities, biological medicines, new indications and repurposed medicines.

The Innovation Passport Application- the first step in the Innovative Licensing and Application Pathway

The first step in the ILAP is the Innovation Passport application.

  • The Innovation Passport is the mandated entry point to the ILAP
  • It is open to developers at the pre-clinical trial stage through to the mid-development programme point.
  • The passport includes a broad and inclusive definition of innovation and both new and repurposed medicines are within scope. This designation is linked to a portfolio of activities through the creation of a product-specific Target Development Profile (TDP).
  • The evidence required for a product to fulfil the criteria will depend on where in the development pathway the product is and can be based on non-clinical data.
  • The Innovation Passport does not replace the Promising Innovation Medicine (PIM) Designation of the EAMS and applicants can apply for both.
  • An applicant is required to submit an Innovation Passport application for each separate medicinal product.

More information here

What are the criteria for the Innovation Passport?

The criteria for the passport are provided below. Each criteria has its own data requirements, available here.

Criteria 1: details of the condition, patient or public health area

a) The condition is life-threatening or seriously debilitating or

b) there is a significant patient or public health need

This is not necessarily linked to the product as it sets out the grounds for the need to develop a medicinal product in a particular area.

Criteria 2: the medicinal product fulfils one or more of the following areas:

a) Innovative medicine such as an advanced therapy medicinal product (ATMP) or new chemical or biological entity or novel drug device combination

b) The medicine is being developed in a clinically significant new indication

c) Medicine for rare disease and/or other special populations such as neonates and children, elderly and pregnant women

d) Development aligns with the objectives for UK public health priorities such as the Chief Medical Officer, Department of Health and Social Care (DHSC) or Life Sciences Sector Deal (including those in Devolved Administrations, where appropriate).

Criteria 3 the medicinal product has the potential to offer benefits to patients

For this criterion the applicant is expected to provide a summary of how patients are likely to benefit from the product or indication coming to market, including:

  • proposed improved efficacy or safety
  • contribution to patient care or quality of life, as compared to alternative therapeutic options. This should be based on evidence from the applicant with the product.
What is the Target Development Profile (TDP)?
  • The Target Development Profile step can only be accessed via the Innovation Passport
  • The Innovation Passport holder should complete a TDP submission form. 
  • The TDP mirrors the target product profile approach. A product-specific team of experts will help define the “target development profile” (TDP) based on the product’s characteristics.
  • It will define key regulatory and development features, identify potential pitfalls and create a road map for delivering early patient access.
  • It will include how the Innovation Passport holder can work together with other UK stakeholders for coordinated and efficient evidence generation and evaluation and address commercial and managed access considerations.
  • It is expected that the TDP will be a living document, updated along the development programme timelines and milestones as new knowledge is generated.
  • For products entering the ILAP at an early stage, there will be multiple TDP versions as data are generated with the product.

The Target Development Profile Toolkit is available here.

What tools will ILAP utilise?

It will utilise the following tools:

  • Adaptive inspections
  • Certifications
  • Continuous Benefit Risk Assessment integrating Real World Evidence (RWE)
  • Clinical Practice Research Datalink (CPRD) Assisted Patient Recruitment
  • Enhanced patient engagement
  • Innovative and Flexible Licensing Routes
  • Novel Methodology and Innovative Clinical Trial Design
  • Rapid Clinical Trial Dossier Pre-Assessment
  • Optimising the Market Access Approach – ILAP HTA Access Tool

More information on the tools is available in this post and/or on the MHRA website here.

What are the benefits of each of the above tools?

Some of the benefits of each tool are detailed below:

Adaptive inspections
  • This tool will support the non-clinical, clinical and manufacturing design and development pathway, to ensure protection of patients and reliability of results.This can therefore be used by developers in support of the authorisation application and onwards to post-authorisation patient supply.
  • Patient benefits include assurance that products that come through the pathway are approved based on valid and reliable data, and that the developer has the necessary infrastructure to support risk identification and management.
  • Applicants can apply to have packages of CTD data (including quality, non-clinical, clinical and pharmacovigilance aspects) assessed by an MHRA multi-disciplinary team. This includes licensing, pharmacovigilance and the inspectorate who will review the submission against marketing authorisation requirements and expectations for regulatory compliance.
  • Such early engagement on the acceptability of the submission will provide the developer with the opportunity to make timely changes to their programme in advance of a formal marketing authorisation application.
  • It is important to note that this tool is not part of a formal marketing authorisation application but provides developers with expert advice on the extent of regulatory readiness of a particular part of the development.
  • For biological medicines, such as vaccines, blood products and other immunological biotherapeutics, applicants will be able to engage early on with the National Institute for Biological Standards and Control (NIBSC) scientists
Continuous Benefit Risk Assessment integrating Real World Evidence (RWE)

Through this tool, MHRA will provide regulatory science expertise,

  • building on the TDP to support activities in the Risk Management Plan (RMP)
  • offering guidance and assistance around development of an upfront data collection and delivery plan.

MHRA will explore with the applicant the options for long-term data collection.

  • This service represents a fast, targeted and efficient approach to patient recruitment across the UK, including the ability to geolocate patients to clinical sties, or inform site location based on areas of highest prevalence.
  • Patient lists can be refreshed on a daily basis and searches can be modified in near real time. A single standardised search of CPRD’s EHR database removes variability of searches and burden on individual GP practices.
Enhanced patient engagement
  • This tool is a key component of the Agency’s broader commitment to be a patient and public-focused regulator i.e. putting patients and the public at the centre of regulatory decision-making.
  • Effective patient engagement offers substantial benefits for companies from providing valuable insight and data, to ensuring innovative products meet patients’ actual medical needs. It also allows patients to add their perspectives on the benefits and risks of medicines.
Innovative and Flexible Licensing Routes
  • Regulatory licensing flexibilities can be used to expedite the approval process once a product or new indication has sufficient data for regulatory review.
  • Independent laboratory testing can also support authorisations based on reduced data packages through the generation, post-licensure, of data supporting batch-to-batch consistency.
Novel Methodology and Innovative Clinical Trial Design
  • This tool has the potential to significantly accelerate development and bring safe, effective medicines to patients faster, balancing the risks and benefits of any novel approaches, with a strong patient focus.
  • Novel trial designs with virtual and decentralised elements will increase patient-centricity in trials and reduce the burden of taking part, improving recruitment and retention.
  • Qualification and multi-stakeholder regulatory acceptance of novel methodologies will de-risk and reduce the costs of development.
Rapid Clinical Trial Dossier Pre-Assessment

This tool:

  • will provide developers with expert feedback on draft submission documentation so that the clinical trial authorisation (CTA) application dossier can be finalised and any sponsor internal sign-offs quickly obtained prior to formal submission
  • will help to reduce the chance of a ‘Grounds for Non-acceptance’ being issued for the trial following official submission and therefore de-risk development. This will also facilitate a quicker trial set-up and development.

More information on the following aspects of of these tools is available here.

  • Tool description
  • Tool benefits
  • What to expect when using this tool
  • Tool delivery partners
  • Related tools and relationship description
What future tools will be available in the toolkit?

The following tools are currently being developed and will be added to the TDP toolkit during 2021:

  • Advanced Therapy Medicinal Product (ATMP) Centre Accreditation
  • Coordinated approval of research involving investigation of both a medicine and medical device
  • Combined in vitro diagnostic (IVD) and investigational medicinal product development
  • CPRD Control Groups
  • Development of quality standards

More information on each of the above tools is available here.

Source: MHRA website

28 Feb 2022Section entitled What tools will ILAP utilise? updated to include the ILAP HTA Access Tool.
24 Jan 2022MHRA website updated information info about MHRA submissions portal
6 Jan 2022MHRA has added information about Fees to its website. The fee is not refundable if your application is not successful.
16 Dec 2021Added a new section entitled Which products are in scope of the ILAP?
5 Oct 2021Changes made to reflect the All Wales Therapeutics and Toxicology Centre (AWTTC) becoming a full partner in the Innovative Licensing and Access Pathway.
23 Aug 2021 Further information added on the MHRA website regarding governance of the ILAP, including operational details of the steering group and patient reference group.
13 Apr 2021 MHRA published Target Development Profile Toolkit for the ILAP licensing route.
27 Feb 2021 First Innovation Passport awarded under MHRA Innovative Licensing and Access Pathway (ILAP)

Source: MHRA website